1. Morquio A: Endurance

2. Endurance testing assesses functional capacity Endurance tests measure the efficiency of performing a task and provide assessments of: Functional status of the cardiac, respiratory and musculoskeletal systems Overall disease progression Quality of life Response to treatments McDonald et al, J Pediatr Rehabil Med, 2010; Hendriksz et al, J Inherit Metab Dis, 2012

3. Image courtesy of Elizabeth Wright Recommended endurance tests for assessing submaximal functional capacity in Morquio A patients include: 6 minute walk test (6MWT) measures how far a person can walk on a hard, flat surface in 6 minutes a standardized test (American Thoracic Society, 2002) used to assess endurance in patients with MPS VI (Harmatz et al, 2005) and Morquio A (Harmatz et al, 2013) 3 minute stair climb (3MSCT) measures how many steps a person can ascend in 3 minutes, using rails and resting as needed used to assess endurance in patients with MPS VI (Harmatz et al, 2005) and Morquio A (Harmatz et al, 2013) Harmatz et al, Mol Genet Metab, 2013; Harmatz et al, Pediatrics, 2005; McDonald et al, J Pediatr Rehabil Med, 2010; Hendriksz et al, J Inherit Metab Dis, 2012 Submaximal intensity tests are sensitive indicators of endurance for MPS patients

4. Endurance is impaired in Morquio A Mean 6MWT = 212.6 ±152.2 m (n = 316 subjects) Mean 3MSCT = 30.0 ±24.0 stairs (n= 274 subjects) Harmatz et al, Mol Genet Metab, 2013 MorCAP baseline data Mean 6MWTMean 3MSCT n=248n=68n=316 n=215n=59n=274

5. Assessment At diagnosis Annually As clinically indicated Evaluation by physiotherapistXXX 6 MWT (if physically and developmentally able)XXX 3 MSCT (if physically and developmentally able)XXX Regular assessments are recommended for optimal patient quality of life Hendriksz et al, J Inherit Metab Dis, 2012